lepunafusp alfa   Click here for help

GtoPdb Ligand ID: 11773

Synonyms: JR-171 | JR171
Compound class: Antibody
Comment: Lepunafusp alfa (JR-171) is a blood-brain-barrier-penetrating form of recombinant human alpha-L-iduronidase (IDUA; P35475), an enzyme that is deficient in patients with the lysosomal storage disease mucopolysaccharidosis type I (MPS I; Hurler syndrome; Scheie syndrome) which leads to disrupted glycosaminoglycan catabolism [1-2]. The enzyme is fused to the same anti-transferrin receptor antibody Fab as pabinafusp alfa, to facilitate movement of the enzyme across the blood-brain barrier. Both lepunafusp and pabinafusp were developed using JCR Pharmaceuticals' proprietary J-Brain Cargo® technology [3].
Selectivity at other protein targets
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Target Sp. Type Action Value Parameter Concentration range (M) Reference
transferrin receptor (CD71) Hs Antibody Binding - - - 3